Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2025 on request of the Sponsor.
This medicine was designated as an orphan medicine for the treatment of medullary thyroid carcinoma in the European Union on 19 February 2021.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:
This medicine, also known as zeteletinib, acts by blocking the activity of a protein called RET, which normally plays a role in the development of several organs. Many patients with medullary thyroid cancer have mutations (changes) in the gene for RET. These mutations cause RET to be activated in an uncontrolled manner, which in turns makes thyroid parafollicular cells multiply out of control and become cancerous. By blocking the activity of RET, the medicine is expected to slow down the growth of the cancer.
Based on description provided by sponsor
At the time of submission of the application for orphan designation:
More information on how potential new medicines are tested during their development is available on Authorisation of medicines.
Medicines intended for rare diseases can be granted an orphan designation during their development.
The orphan designation allows the developer to benefit from:
To qualify for orphan designation, a medicine must meet a number of criteria:
EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.
The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
For more information, see:
Propharma Group The Netherlands B.V.
| Date | Update |
|---|---|
| September 2023 | The sponsorship was transferred from Southwood Research Ltd., Netherlands to Propharma Group The Netherlands B.V., Netherlands. |
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: